Publications by authors named "N Remus"
Gathering real-world compassionate data to expand eligibility for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with N1303K or other rare variants: a viewpoint.
Eur Respir J
January 2024
Article Synopsis
- * A study analyzed data from 174 CF patients with severe lung impairment (FEV ≤ 40%), finding that lower peak work rate (W) and peak oxygen uptake were significant predictors of death/LTX.
- * The research revealed that patients with a peak work rate of 49.2% or lower had a much higher risk (45.2%) of death/LTX compared to those above that threshold (10.9%), indicating that W could be crucial for deciding on transplant referrals.
Lower respiratory tract infections (LRTI) encompass a wide range of clinical syndromes, prominently including bronchiolitis, bronchitis and pneumonia. LRTIs are the second leading cause of antibiotic prescriptions. The vast majority of these infections are due to (or triggered by) viruses and are self-limited diseases.
View Article and Find Full Text PDFBackground: Around 20% of people with cystic fibrosis (pwCF) do not have access to the triple combination elexacaftor/tezacaftor/ivacaftor (ETI) in Europe because they do not carry the F508del allele on the CF transmembrane conductance regulator () gene. Considering that pwCF carrying rare variants may benefit from ETI, including variants already validated by the US Food and Drug Administration (FDA), a compassionate use programme was launched in France. PwCF were invited to undergo a nasal brushing to investigate whether the pharmacological rescue of CFTR activity by ETI in human nasal epithelial cell (HNEC) cultures was predictive of the clinical response.
View Article and Find Full Text PDFBackground: The European Medicines Agency has approved the cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination elexacaftor/tezacaftor/ivacaftor (ETI) for people with cystic fibrosis (CF) carrying at least one F508del variant. The United States Food and Drug Administration (FDA) also approved ETI for people with CF carrying one of 177 rare variants.
Methods: An observational study was conducted to evaluate the effectiveness of ETI in people with CF with advanced lung disease who were not eligible for ETI in Europe.